On July 20, 2022 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported new regulatory designations for eltanexor, a novel oral, Selective Inhibitor of Nuclear Export (SINE) investigational compound being studied for the treatment of myelodysplastic syndromes (MDS): (i) the U.S. Food and Drug Administration (FDA) has granted fast track designation for the development program of eltanexor as monotherapy for the treatment of patients with relapsed or refractory intermediate, high-, or very high-risk MDS; (ii) the European Commission (EC) adopted the Committee for Orphan Medicinal Products (COMP) opinion to designate eltanexor as an orphan medicinal product for the treatment of MDS in the European Union (EU) (Press release, Karyopharm, JUL 20, 2022, View Source [SID1234616787]). Karyopharm also received orphan drug designation from the FDA in January 2022. MDS are a group of diseases characterized by ineffective production of the components of the blood due to poor bone marrow function with a risk of progression to acute myeloid leukemia.

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Karyopharm is currently investigating eltanexor in an ongoing open-label Phase 1/2 study in patients with relapsed/refractory MDS. Previously, Karyopharm reported initial data from the Phase 1 portion of this study evaluating single-agent eltanexor in patients with hypomethylating agent (HMA)-refractory MDS.

Approximately 15,000 people in the U.S.1 and 14,000 people in the EU2 are expected to be diagnosed with intermediate-to-high risk MDS in 2022. HMAs are the current standard of care for newly diagnosed, higher-risk MDS patients. However, only 40-60% of patients respond, with these responses typically lasting less than two years.3 The prognosis in HMA-refractory disease is poor, with a median overall survival of four to six months.4,5 There are currently no approved therapies for HMA- refractory MDS.

"These recent designations from the FDA and EC reinforce eltanexor’s potential to improve clinical outcomes for patients with relapsed/refractory MDS," said Richard Paulson, President and Chief Executive Officer of Karyopharm. "We are dedicated to advancing our ongoing clinical trials and remain committed to bringing eltanexor to these patients and their families as a new treatment option."

Fast track is a process designed by the FDA to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious conditions. Once a drug receives Fast Track designation, early and frequent communication between the FDA and the drug company is encouraged throughout the entire drug development and review process.

Orphan Medicinal Product Designation is granted by the EC to promote the development of drugs that target rare (less than 5 in 100,000 people across the EU), seriously debilitating and/or life-threatening diseases, and are expected to provide a significant benefit over existing authorized treatments. Orphan designation qualifies a company for certain incentives that apply across all stages of drug development, including the potential for ten years of market exclusivity following marketing approval, fee reductions, and eligibility for orphan drug grants.

About Eltanexor

Eltanexor (KPT-8602) is an investigational novel SINE compound that functions by binding with, and inhibiting, the nuclear export protein, XPO1, leading to the accumulation of tumor suppressor proteins in the cell nucleus. This reinitiates and amplifies their tumor suppressor function and is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells.

In preclinical models, eltanexor has a broad therapeutic window with minimal penetration of the blood brain barrier and, therefore, has the potential to serve as another SINE compound for cancer indications. Following oral administration, animals treated with eltanexor show lower percentage of body weight loss and improved food consumption than animals similarly treated with selinexor. This allows more frequent dosing of eltanexor, enabling a longer period of exposure than is possible with selinexor.

Eltanexor is an investigational medicine and has not been approved by the United States Food and Drug Administration or any other regulatory agency.